Head of FDA’s Rare Disease Division Departs, Leaving Agency Looking for New Leader

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The US Food and Drug Administration (FDA) is looking for a new leader for its Rare Diseases Program. In an email to staff on 16 July 2014, John Jenkins, director of the Office of New Drugs (OND), which oversees the rare disease program, said the […]

BioCentury Presents: Right To Try Laws

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TATES’ RIGHTS BY STEVE USDIN, WASHINGTON EDITOR State right-to-try laws, which have been signed by a Democratic governor in Colorado and a Republican in Louisiana, are responses to deep dissatisfaction with the pace of drug development and frustration with the ways biopharma companies have responded […]

Boehringer Ingelheim Announces Nintedanib Expanded Access Program (EAP) for Patients with Idiopathic Pulmonary Fibrosis

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Boehringer Ingelheim Pharmaceuticals, Inc. today announced that the company has initiated an open-label, U.S. multi-center expanded access program (EAP) for its investigational compound nintedanib, for people diagnosed with idiopathic pulmonary fibrosis (IPF) and who meet certain eligibility criteria. Nintedanib is an investigational therapy and has not […]

Registry for Fatal Lung Disease Aims to Speed Improvements in Care

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Duke Clinical Research Institute (DCRI) has launched a patient registry to help researchers and clinicians identify, manage and study people who have a progressive lung disease called idiopathic pulmonary fibrosis. The registry, a joint effort by DCRI and Boehringer Ingelheim Pharmaceuticals, Inc., is the first […]

CureDuchenne Makes Statement Regarding Prosensa Announcement of Regulatory Path Forward for Drisapersen as a Potential Treatment for DMD

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CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, issued the following statement today regarding the announcement by Prosensa Holding N.V. (NASDAQ: RNA) that the United States Food and Drug Administration (FDA) has outlined a regulatory path […]

Register Now! June 10th FDA Patient-Focused Disease-Specific Meeting

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FDA Public Meeting information   Title: “Neurological Manifestations of Inborn Errors of Metabolism”   Goal: To obtain patient input on the impact of the neurological manifestations of inborn errors of metabolism on daily life and patient views on treatment options.   Date: June 10, 2014 […]

Patients Benefit from Open Dialogue and Scientifically Accurate Information on Medicines

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Some draft guidances issued by the Food and Drug Administration (FDA) have a more direct impact on patients than others. The Agency’s recent draft guidance on social media could have a direct impact on the ability of me and other patients to get information on […]